Duchenne muscular dystrophy’s progress so far in 2023

DMD Awareness Week took place from 13 to 19 February this year. Santhera Pharmaceuticals and ReveraGen BioPharma announced on 9 January that the US Food and Drug Administration (FDA) had accepted their new drug application (NDA) for vamorolone, a dissociative delta-9, 11 glucocorticoid analogue, a steroidal drug that targets NR3C1. The Prescription Drug User Fee Act (PDUFA) date is set for 26 October this year. Santhera and ReveraGen are using the positive results from the pivotal Phase IIb VISION-DMD study in support of the NDA. Vamorolone currently has orphan drug, fast track and rare paediatric disease designations.